Amyotrophic lateral sclerosis and gene therapy

Abstract

With the identification of the genes and proteins involved in the etiology of neurodegenerative diseases, and the development of new viral tools for gene delivery, modulating the function of disease-associated genes as a therapy has become an increasingly realistic possibility. This approach is particularly applicable to amyotrophic lateral sclerosis (ALS), the dominant clinical manifestation of which is weakness that progresses to paralysis, with ensuing respiratory failure and death within 3–5 years of onset. A significant proportion of familial ALS cases are caused by a dominant mutation in the gene encoding superoxide dismutase (SOD1) that confers an aberrant toxic property on the protein; in principle, these cases could be amenable to treatment employing a gene-silencing strategy.

HPAN: A BioMed 21 IRC

The Hope Center Program on Protein Aggregation and Neurodegeneration (HPAN) is the most formalized of the Hope Center Research Groups. HPAN, led by Director David Holtzman, is one of five Interdisciplinary Research Centers (IRCs) based in the new BJC Institute of Health. This group focuses on the shared mechanism of protein aggregation that underlies numerous neurological disorders. Read more about HPAN....